Intellia Therapeutics on Wednesday said it has received a green light from the Food and Drug Administration to start a Phase 3 trial of a gene editing treatment it’s developing for the rare disease transthyretin amyloidosis.

周三Intellia Therapeutics表示，美国食品和药物管理局已经发出绿灯，开始进行基因编辑治疗的3期临床试验，该治疗正在开发罕见疾病转甲状腺素蛋白淀粉样变性。

According to Intellia, the trial will be the first late-stage test in the U.S. of a CRISPR-based therapy that’s works in vivo, or by editing genes inside the human body rather than in cells extracted from patients. Intellia expects to begin the study by the end of the year.

根据Intellia的说法，该试验将是美国第一个在体内起作用的基于CRISPR的疗法的晚期试验，或者通过编辑人体内的基因而不是从患者体内提取的细胞来进行。Intellia预计在年底之前开始研究。

Dubbed NTLA-2001, Intellia’s therapy is designed to be a one-time fix for transthyretin amyloidosis. Using CRISPR/Cas9, it knocks out a gene responsible for producing proteins that, in people with the condition, are damaged and build up in toxic accumulations in the heart and nerves.

Intellia的疗法被称为NTLA-2001，旨在一次性解决运甲状腺素蛋白淀粉样变性。使用CRISPR/Cas9，它敲除了一种基因，该基因负责产生蛋白质，这些蛋白质在患有这种疾病的人中会受到损害并在心脏和神经中积聚有毒物质。

Intellia developed NTLA-2001 together with Regeneron, with which it’s worked since 2016. The companies recently expanded their collaboration to develop in vivo medicines for neurological and muscular diseases.

Intellia与Regeneron一起开发了NTLA-2001，自2016年以来一直与其合作。这些公司最近扩大了合作，开发用于神经和肌肉疾病的体内药物。

A few medicines are approved in the U.S. to treat nerve damage caused by transthyretin amyloidosis. One, from Pfizer, is cleared for the cardiomyopathy form of the disease, which is the target of Intellia’s Phase 3 study. (Intellia is also planning another study in the polyneuropathy disease type.)

美国批准了一些药物来治疗由转甲状腺素蛋白淀粉样变性引起的神经损伤。辉瑞公司（Pfizer）的一家公司被批准用于该疾病的心肌病形式，这是Intellia 3期研究的目标。（Intellia还计划在多发性神经病类型中进行另一项研究。）

The go-ahead for Intellia to start its Phase 3 trial signals increasing confidence from the FDA in CRISPR gene editing treatments, analysts said.

分析师表示，Intellia开始其3期试验的前进标志着FDA对CRISPR基因编辑治疗的信心日益增强。

“Today's [Investigational New Drug application] clearance signals that [the] FDA is comfortable with the potential risks of off-target editing related to in vivo gene editing therapies and provides further regulatory derisking for [Intellia’s] platform,” wrote Kostas Biliouris, an analyst at BMO Capital Markets..

BMO资本市场分析师Kostas Biliouris写道：“今天的[研究性新药申请]清除信号表明[FDA]对与体内基因编辑疗法相关的脱靶编辑的潜在风险感到满意，并为[Intellia]平台提供了进一步的监管推导。。

Emerging clinical data may have also bolstered Intellia’s case, Biliouris said.

Biliouris说，新兴的临床数据也可能支持Intellia的案例。

Other in vivo gene editing treatments have been cleared for U.S. trials, including ones from Sangamo Biosciences, Excision BioTherapeutics and another from Intellia. NTLA-2001 is the furthest along in testing, however.

其他体内基因编辑治疗已被美国试验批准，包括来自Sangamo Biosciences，Excision BioTherapeutics和Intellia的试验。然而，NTLA-2001是测试中最远的。

“Successful execution here affirms [Intellia] as the front-runner among companies … working to commercialize in vivo editing,” Mani Foroohar, an analyst at Leerink Partners, wrote in a note to clients.

Leerink Partners的分析师Mani Foroohar在致客户的笔记中写道：“这里的成功执行肯定[Intellia]是公司的领先者……致力于体内编辑的商业化。

The green light from the FDA comes as Vertex Pharmaceuticals and CRISPR Therapeutics await the FDA’s verdict on approval of an ex vivo treatment for sickle cell disease. If approved by the agency’s Dec. 8 target, it would be the first CRISPR-based medicine to arrive on market in the U.S.

来自FDA的绿灯来自Vertex Pharmaceuticals和CRISPR Therapeutics等待FDA关于批准镰状细胞病离体治疗的判决。如果得到该机构12月8日目标的批准，这将是第一个在美国上市的基于CRISPR的药物。

Shares in Intellia dropped by about 4% in Wednesday morning trading.

在周三早上交易中，Intellia的股票下降了约4%。